Medunik Canada, is an orphan drug company dedicated to helping Canadians with rare diseases access therapies currently unavailable in Canada, has signed an exclusive distribution agreement with Orphan Europe Recordati Group.

Under this agreement, Medunik Canada receives the exclusive Canadian rights to market and distribute four important therapies in the following medical conditions: (1) acute hepatic porphyria, (2) hyperammonaema due to N-acetylglutamate synthase (NAGS) deficiency or one of three organic acidurias (isovaleric, methylmalonic or propionic), (3) patent ductus arteriosus and (4) vitamin E deficiency in chronic cholestasis.

“We are thrilled to enter into a new partnership with Orphan Europe Recordati Group” stated Éric Gervais, executive vice-president of Medunik Canada. “This new strategic collaboration complements our existing alliances and represents an important milestone in our endeavour to help patients with a rare disease in Canada to benefit from the best treatments available”.

Marco Liguori, general manager & Recordati vice-president, special care and orphan drugs went on to say that “Orphan Europe Recordati Group is delighted to extend its products and services to rare disease patients in Canada. We are particularly happy to work with a company like Medunik Canada which is committed to building awareness and promoting better diagnosis and care of rare disease patients in Canada.”

Acute hepatic porphyrias: The porphyrias are a group of disorders of the heme biosynthesis pathway that present with acute neurovisceral symptoms, skin lesions or both. Acute porphyria is a term that includes three similar inherited diseases: acute intermittent porphyria (AIP), variegate porphyria (VP) and hereditary coproporphyria (HCP). They are characterised by episodic acute neurovisceral attacks which may be life threatening. These attacks are uncommon and are often difficult to diagnose. In AIP only acute attacks occur and the skin is never affected, whilst in VP or HCP skin may also be affected.

Hyperammonaema due to N-acetylglutamate synthase (NAGS) deficiency or one of three organic acidurias (isovaleric, methylmalonic or propionic): NAGS deficiency is one of the urea cycle disorders (UCD). UCDs are caused by a deficiency of one of the enzymes in the urea cycle which is responsible for removing ammonia from the blood stream and organic acidurias disrupt normal amino acid metabolism causing a build-up of acids. Both groups of disorders can cause hyperammonemia. Accumulation of ammonia in the body causes irreversible brain damage, coma, and can be fatal.

Patent Ductus Arteriosus (PDA): In the developing fetus, the ductus arteriosus (DA) is a shunt connecting the pulmonary artery to the aortic arch. It allows the blood from the right ventricle to bypass the lungs. In normal newborns, the DA is closed within a few hours after birth. However, in preterm babies, this blood vessel sometimes remains open and because of the pressure gradient blood flows back from the aorta to the pulmonary artery. This condition is called patent ductus arteriosus (PDA) and affects the perfusion of the lungs and other organs (brain, kidney, mesentery).

Vitamin E deficiency in chronic cholestasis: Chronic cholestasis represents a symptom of various conditions where the flow or the formation of bile is impaired. Patients with chronic cholestasis cannot absorb fat soluble vitamins. Vitamin E deficiency is therefore a complication often observed in patients presenting with this condition and can lead to neurological abnormalities.

Orphan Europe is a pharmaceutical company aiming at providing treatment for patients with unmet medical needs suffering from rare diseases. Being part of Recordati Group since December 2007, it continues to be one of the most active players in the field of rare diseases.